公开(公告)号：US20200069817A1

公开(公告)日：2020-03-05

申请号：US16537192

申请日：2019-08-09

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Tongyao LIU ,  Alexey SEREGIN ,  Robert T. PETERS ,  Jiayun LIU ,  Philip ZAKAS ,  Douglas DRAGER ,  Susannah PATARROYO-WHITE

IPC: A61K48/00 ,  C12N15/86 ,  C12N7/00 ,  C07K14/755 ,  A61P7/04 ,  A61K9/00

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the target sequence encodes a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, a growth factor, a hormone, a cytokine, an antibody, a fragment thereof, and a combination thereof. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating a metabolic disorder of the liver in a subject comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.

公开(公告)号：US20210163986A1

公开(公告)日：2021-06-03

申请号：US16636583

申请日：2018-08-09

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Alexey SEREGIN ,  Tongyao LIU ,  Susannah PATARROYO-WHITE ,  Douglas DRAGER ,  Robert T. PETERS ,  Jiayun LIU

IPC: C12N15/86 ,  C07K14/755

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, e.g., a FVIII polypeptide, a FIX polypeptide, or a fragment thereof. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.